Extract
CFTR modulators have revolutionized cystic fibrosis (CF) care, leading to significant improvements in lung function, decreased frequency of pulmonary exacerbations and improved quality of life [1]. The list of variants eligible for CFTR modulators has increased overtime, currently covering almost 90% of individuals with CF in Caucasian populations. For rare and poorly characterized CF-causing variants, ex vivo analysis of intestinal organoids may assess CFTR function and guide personalised therapy [2, 3]. We report the case of an individual with CF whose rectal organoids were used to evaluate response to CFTR modulators and to guide therapeutic decision.
Footnotes
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Conflict of Interest: GM, ZB, SB, GD, AS report no financial relationships pertaining to this work.
Conflict of Interest: NB, SH, CC have received funding for organoid research from FreeNovation (a funding program of the Novartis Research Foundation), the Swiss Society for Cystic Fibrosis (CFCH), the Swiss Foundation “Respirer” and EIT Health, a body receiving support from the European Union's Horizon 2020 Research and innovation program.
Conflict of Interest: NB and SH have leadership roles in SUN bioscience S.A. The Ecole Polytechnique Fédérale de Lausanne has filed for patent protection on technologies enabling organoid assays, and ND and SH are named as inventor on those patents; NB and SH are shareholders in SUN bioscience SA, which is commercialising those patents.
Conflict of Interest: AK has received the Grant Pépinière from the University of Lausanne, a Research Grant from the Swiss Lung Association (grant number 2018-16), and a BRIDGE Discovery Grant (40B2-0_194701/1) from the Swiss National Science Foundation (SNFS).
- Received July 5, 2022.
- Accepted October 31, 2022.
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